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WASHINGTON — A drug to treat the most life-threatening forms of high cholesterol has won U.S.
approval and will carry a label warning about the risk of liver toxicity.

The Food and Drug Administration cleared the treatment known as lomitapide for use in patients
with a rare genetic disease that causes extreme elevation of blood-cholesterol levels, maker
Aegerion Pharmaceuticals Inc. said yesterday. The drug is the company’s first on the market.

The once-a-day pill called Juxtapid is designed to treat homozygous familial
hypercholesterolemia, a condition that causes abnormalities in liver cells responsible for clearing
LDL — so-called bad cholesterol — from the blood. The disease can lead to heart attack or death at
an early age, according to the National Institutes of Health.

“We are excited that Juxtapid will become a new treatment option for patients with HoFH,” said
Marc Beer, chief executive officer at Aegerion, which is based in Cambridge, Mass.

Juxtapid will carry a boxed warning, the FDA’s strictest caution, alerting patients to the risk
of liver toxicity. Of the 29 patients in a clinical trial, 34 percent had at least one elevation in
an enzyme that indicates liver damage, according to the boxed warning.

The market for the treatment could total as many as 3,000 Americans, Beer said. The drug will
cost between $200,000 and $300,000 per patient per year, he said.

When added to existing lipid-lowering therapy, Juxtapid helped reduce LDL cholesterol by about
50 percent in a trial of 23 patients after 26 weeks, according to the company.